JP2016519933A5 - - Google Patents

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JP2016519933A5
JP2016519933A5 JP2016513129A JP2016513129A JP2016519933A5 JP 2016519933 A5 JP2016519933 A5 JP 2016519933A5 JP 2016513129 A JP2016513129 A JP 2016513129A JP 2016513129 A JP2016513129 A JP 2016513129A JP 2016519933 A5 JP2016519933 A5 JP 2016519933A5
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genetically engineered
blood cell
enucleated blood
engineered enucleated
erythrocyte
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JP6599848B2 (en
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タンパク質性薬物と第１の赤血球膜タンパク質とを含む第１の融合タンパク質を表面上に発現することを特徴とする遺伝子操作された脱核血液細胞。 A genetically engineered enucleated blood cell characterized by expressing on a surface a first fusion protein comprising a proteinaceous drug and a first erythrocyte membrane protein. 請求項１に記載の遺伝子操作された脱核血液細胞において、前記第１の赤血球膜タンパク質がＩ型赤血球膜貫通型タンパク質であることを特徴とする遺伝子操作された脱核血液細胞。The genetically engineered enucleated blood cell according to claim 1, wherein the first erythrocyte membrane protein is a type I erythrocyte transmembrane protein. 請求項１に記載の遺伝子操作された脱核血液細胞において、前記第１の赤血球膜タンパク質がＩ型赤血球膜貫通型タンパク質であり、かつ前記タンパク質性薬物が前記Ｉ型赤血球膜貫通型タンパク質のＮ末端に融合されていることを特徴とする遺伝子操作された脱核血液細胞。 2. The genetically engineered enucleated blood cell of claim 1, wherein the first erythrocyte membrane protein is a type I erythrocyte transmembrane protein and the proteinaceous drug is N of the type I erythrocyte transmembrane protein. A genetically engineered enucleated blood cell characterized by being fused to the end. 請求項２又は３に記載の遺伝子操作された脱核血液細胞において、前記Ｉ型赤血球膜貫通型タンパク質がグリコフォリンＡ（ＧＰＡ）であることを特徴とする遺伝子操作された脱核血液細胞。 The genetically engineered enucleated blood cell according to claim 2 or 3 , wherein the type I erythrocyte transmembrane protein is glycophorin A (GPA). 請求項１に記載の遺伝子操作された脱核血液細胞において、前記第１の赤血球膜タンパク質がＩＩ型赤血球膜貫通型タンパク質であることを特徴とする遺伝子操作された脱核血液細胞。The genetically engineered enucleated blood cell according to claim 1, wherein the first erythrocyte membrane protein is a type II erythrocyte transmembrane protein. 請求項１に記載の遺伝子操作された脱核血液細胞において、前記第１の赤血球膜タンパク質がＩＩ型膜貫通型タンパク質であり、かつ前記タンパク質性薬物が前記ＩＩ型膜貫通型タンパク質のＣ末端に融合されていることを特徴とする遺伝子操作された脱核血液細胞。 2. The genetically engineered enucleated blood cell of claim 1, wherein the first erythrocyte membrane protein is a type II transmembrane protein and the proteinaceous drug is present at the C-terminus of the type II transmembrane protein. A genetically engineered enucleated blood cell characterized by being fused. 請求項５又は６に記載の遺伝子操作された脱核血液細胞において、前記ＩＩ型赤血球膜貫通型タンパク質がＫｅｌｌまたはＣＤ７１であることを特徴とする遺伝子操作された脱核血液細胞。 The genetically engineered enucleated blood cell according to claim 5 or 6 , wherein the type II erythrocyte transmembrane protein is Kell or CD71. 請求項１乃至７の何れか１項に記載の遺伝子操作された脱核血液細胞において、前記タンパク質性薬物が、ワクチン抗原、癌抗原、病原体に由来する抗原、酵素、または凝固（ｃｌｏｔｔｉｎｇまたはｃｏａｇｕｌａｔｉｏｎ）因子を含むことを特徴とする遺伝子操作された脱核血液細胞。 In enucleated blood cells that have been genetically engineered according to any one of claims 1 to 7, wherein the protein drug is, vaccine antigens, cancer antigens, antigens derived from a pathogen, enzymes or coagulation (clotting or coagulation, ) Genetically engineered enucleated blood cells characterized by containing factors . 請求項１乃至８の何れか１項に記載の遺伝子操作された脱核血液細胞において、前記タンパク質性薬物が抗体またはそのフラグメントであることを特徴とする遺伝子操作された脱核血液細胞。 The genetically engineered enucleated blood cell according to any one of claims 1 to 8 , wherein the proteinaceous drug is an antibody or a fragment thereof. 請求項９に記載の遺伝子操作された脱核血液細胞において、前記抗体が単一ドメイン抗体であることを特徴とする遺伝子操作された脱核血液細胞。 The genetically engineered enucleated blood cell of claim 9 , wherein the antibody is a single domain antibody. 請求項１乃至１０の何れか１項に記載の遺伝子操作された脱核血液細胞において、第２の目的ペプチドと第２の赤血球膜タンパク質とを含む第２の融合タンパク質を表面上にさらに発現することを特徴とする遺伝子操作された脱核血液細胞。 11. The genetically engineered enucleated blood cell according to any one of claims 1 to 10 , wherein a second fusion protein comprising a second target peptide and a second erythrocyte membrane protein is further expressed on the surface. A genetically engineered enucleated blood cell characterized in that. 請求項１乃至１１の何れか１項に記載の遺伝子操作された脱核血液細胞において、遺伝子操作された脱核血液細胞がヒト脱核赤血球であることを特徴とする遺伝子操作された脱核血液細胞。The genetically engineered enucleated blood cell according to any one of claims 1 to 11, wherein the genetically engineered enucleated blood cell is a human enucleated erythrocyte. cell. 請求項１乃至１２の何れか１項に記載の遺伝子操作された脱核血液細胞において、前記第１の赤血球膜貫通型タンパク質が天然のタンパク質またはペプチドのフラグメントであることを特徴とする遺伝子操作された脱核血液細胞。The genetically engineered enucleated blood cell according to any one of claims 1 to 12, wherein the first erythrocyte transmembrane protein is a natural protein or a fragment of a peptide. Enucleated blood cells. 請求項１乃至１３の何れか１項に記載の複数の細胞と、薬学的に許容される担体とを含むことを特徴とする医薬組成物。A pharmaceutical composition comprising the plurality of cells according to any one of claims 1 to 13 and a pharmaceutically acceptable carrier. 医薬組成物において、治療薬を必要とする被験体に治療薬を送達するための複数の細胞であって、請求項１乃至１３の何れか１項に記載の複数の細胞を含むことを特徴とする医薬組成物。A pharmaceutical composition comprising a plurality of cells for delivering a therapeutic agent to a subject in need of the therapeutic agent, comprising a plurality of cells according to any one of claims 1 to 13. A pharmaceutical composition.
JP2016513129A 2013-05-10 2014-05-09 In vitro generation of red blood cells with sortable proteins Active JP6599848B2 (en)

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